Verastem Oncology (NASDAQ: VSTM) has recently announced positive updated safety and efficacy results from the ramp 205 phase 1/2 trial, which is evaluating avutometinib plus defactinib in combination with gemcitabine and nab-paclitaxel in the frontline for patients with metastatic pancreatic ductal adenocarcinoma (PDAC). As of April 25, 2025, patients in the dose level 1 cohort, which was selected as the recommended phase 2 dose (RP2D), achieved an overall response rate (ORR) of 83% (10/12). These strong and consistent results have led the company to select dose level 1 as the RP2D and to advance beyond the first stage of the expansion study, enrolling up to 29 patients at this dose level.
In evaluating all the dose cohorts, dose level 1 demonstrated the highest response rate, and across all five dose cohorts, 92% (48/52) of efficacy evaluable patients showed tumor reduction as the best response. Furthermore, adverse events across all dose cohorts remained generally consistent with the previously announced safety and tolerability profile, with no new safety signals emerging. The company is now developing plans for a registrational phase 3 frontline metastatic PDAC trial to begin in 2026.
Verastem Oncology's President and CEO, Dan Paterson, emphasized the significance of these results, stating that they reinforce the company's commitment to maximizing the synergistic potential of the avutometinib plus defactinib combination in other advanced solid tumors for market expansion opportunities beyond kras-mutated recurrent low-grade serous ovarian cancer.
In addition to the ramp 205 trial, the company will also be reviewing the updated data from the phase 1/2 study in China of VS-7375, an oral KRAS G12D inhibitor, during an R&D investor webcast on Monday, June 2, 2025. The study in China was approved in June 2024, and the first patient was dosed in July 2024.
The combination therapy of avutometinib (AVMAPKI) and defactinib (FAKZYNJA) has received FDA approval for the treatment of adult patients with kras-mutated recurrent low-grade serous ovarian cancer. Verastem is also evaluating avutometinib in combination with defactinib and other agents as a potential treatment for patients with advanced pancreatic cancer and advanced kras G12C mutant non-small cell lung cancer.
The company has provided important safety information and adverse reactions related to the AVMAPKI FAKZYNJA co-pack, and is actively working on plans for a registrational phase 3 frontline metastatic PDAC trial. Today the company's shares have moved -1.71% to a price of $7.48. Check out the company's full 8-K submission here.
